BPC January 07 update

LOXO acquired by Eli - shares +66%; SAGE +43% and AXSM +161% release positive depression data

Price and Volume Movers

Eli Lilly and Company (NYSE: LLY) announced the acquisition of Loxo Oncology, Inc. (NASDAQ: LOXO) for $235 per share in cash, or approximately $8b. The transaction is not subject to any financing condition and is expected to close this quarter. Shares of Loxo closed up 66% to $232.65.

Sage Therapeutics (NASDAQ:SAGE) shares rallied to close up 43% to $139.13 following news that its Phase 3 trial of SAGE-217 in women with postpartum depression (PPD) met the primary endpoint. After two weeks of outpatient treatment, patients treated with SAGE-217 had a statistically significant improvement of 17.8 points in the Hamilton Rating Scale for Depression (HAMD-17) score, compared to 13.6 for placebo (p=0.0029).

Axsome Therapeutics, Inc. (NASDAQ: AXSM) shares surged to close up 161% to $6.87 on news that its Phase 2 trial of AXS-05 for the treatment of major depressive disorder (MDD), met the prespecified primary endpoint and significantly improved symptoms of depression. At Week 6, AXS-05 demonstrated a 17.2 point reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score compared to a 12.1 point reduction for bupropion (p=0.013).

Insmed Incorporated (NASDAQ: INSM) shares closed up 43% to $21.27 following news released late-Friday noting that sales of its recently approved Arikayce hit $9.8m in the latest quarter.

La Jolla Pharmaceutical Company (NASDAQ: LJPC) shares slumped to close down 50% to $5.24 on news that sales of its low blood pressure drug Giapreza are expected to hit $24-28m this year, well below analyst expectations.

AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) and Palatin Technologies, Inc.’s (NYSE: PTN) announced that the PDUFA date for Vyleesi (Bremelanotide) has been extended by three months to June 23, 2019. An FDA-requested frequent-dosing study with premenopausal volunteers assessing short term daily use of Vyleesi has been initiated. Results will be submitted to the FDA prior to the updated PDUFA date of June 23, 2019.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

Bellerophon Therapeutics, Inc. (BLPH): $1.09; +27%.

Reata Pharmaceuticals, Inc. (RETA): $72.75; +23%.

Clovis Oncology, Inc. (CLVS): $21.73; +18%.

Coherus BioSciences, Inc. (CHRS): $10.25; +18%.

Verrica Pharmaceuticals Inc. (VRCA): $11.54; +16%.

DECLINERS:

Collegium Pharmaceutical, Inc. (COLL): $15.06; -20%.

Translate Bio, Inc. (TBIO): $7.32; -13%.

scPharmaceuticals Inc. (SCPH): $3.37; -12%.

Cocrystal Pharma, Inc. (COCP): $3.74; -9%.

vTv Therapeutics Inc. (VTVT): $2.37; -9%.

Pipeline updates below: 

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADMA – ADMA Biologics Inc
BIVIGAM
Primary humoral immunodeficiency

CRL Response to Complete Response Letter submitted January 7, 2019.
$149.3 million

ADMS – Adamas Pharmaceuticals Inc.
GOCOVRI (amantadine)
Multiple sclerosis (MS)

Phase 3 Phase 3 data due 2H 2019.
$235.4 million

ADRO – Aduro Biotech Inc.
ADU-S100 and Spartalizumab
Solid tumors or lymphomas

Phase 1b Phase 1 dose escalation data due 2H 2019.
$204.9 million

ADVM – Adverum Biotechnologies Inc.
ADVM-022
Wet age-related macular degeneration (Wet-AMD)

Phase 1 Phase 1 trial initiation announced November 19, 2018. Interim data due 1Q 2020.
$191.7 million

AGIO – Agios Pharmaceuticals Inc.
AG-348 - Mitapivat
Thalassemia

Phase 2 Phase 2 data due 2H 2019.
$3 billion

AGIO – Agios Pharmaceuticals Inc.
Ivosidenib and Vorasidenib (AG-881)
IDHm low-grade glioma

Phase 1 Phase 1 data due 1H 2019.
$3 billion

AGIO – Agios Pharmaceuticals Inc.
Ivosidenib
Frontline AML with IDH1 mutation

sNDA Filing sNDA filed 4Q 2018.
$3 billion

AGIO – Agios Pharmaceuticals Inc.
Vorasidenib (AG-881)
Glioma

Phase 3 Phase 3 trial to be initiated YE 2019.
$3 billion

AGIO – Agios Pharmaceuticals Inc.
AG-270
Solid tumors

Phase 1 Phase 1 dose escalation data due 1H 2019.
$3 billion

AGIO – Agios Pharmaceuticals Inc.
AG-120 Ivosidenib
IDH1 mutant positive cholangiocarcinoma - cancer

Phase 3 Phase 3 top-line data due 1H 2019.
$3 billion

AGN – Allergan plc
Bimatoprost
Open-angle glaucoma or ocular hypertension

NDA Filing NDA filing due 2H 2019.
$54.2 billion

AGRX – Agile Therapeutics Inc.
Twirla
Contraceptive patch

NDA Filing CRL issued 2013. Further CRL issued December 22, 2017. NDA to be refiled 1H 2019 following completion of comparative wear study 1Q 2019.
$30.6 million

ALKS – Alkermes plc
Aristada and Invega Sustenna
Schizophrenia

Phase 3 Phase 3 data due 1H 2019.
$5 billion

ALKS – Alkermes plc
ALKS 3831 - ENLIGHTEN-Early
Schizophrenia - young adults

Phase 3 Phase 3 data due early 2020.
$5 billion

ALLK – Allakos Inc.
AK002
Chronic Urticaria

Phase 2 Phase 2 data released January 7, 2019. 12 of 13 CRs.
$1.8 billion

AMAG – AMAG Pharmaceuticals Inc.
Vyleesi (Bremelanotide)
Female sexual dysfunction (FSD)

PDUFA PDUFA date extended to June 23, 2019.
$578.6 million

ARDX – Ardelyx Inc.
Tenapanor
Hyperphosphatemia in end-stage renal disease (ESRD) patients

Phase 3 Phase 3 released February 15, 2017 - primary endpoint met. Data from second Phase 3 trial due 4Q 2019.
$120.5 million

ARNA – Arena Pharmaceuticals Inc.
Etrasimod
Atopic dermatitis (AD)

Phase 2 Phase 2 trial to be initiated 2019.
$2.1 billion

ARNA – Arena Pharmaceuticals Inc.
Olorinab (APD371)
Pain associated with Crohn's disease

Phase 2 Phase 2/3 trial to be initiated 2019.
$2.1 billion

ARNA – Arena Pharmaceuticals Inc.
Etrasimod
Ulcerative colitis

Phase 2 Phase 3 trial to be initiated 2019.
$2.1 billion

ARWR – Arrowhead Pharmaceuticals Inc.
ARO-ANG3
Dyslipidemia

Phase 1 Phase 1 initiation announced January 7, 2019.
$1.2 billion

ARWR – Arrowhead Pharmaceuticals Inc.
AROAPOC31001
Familial chylomicronemia syndrome (FCS)

Phase 1 Phase 1 trial to be initiated 2019.
$1.2 billion

ASMB – Assembly Biosciences Inc.
ABI-H0731
Hepatitis B virus (HBV)

Phase 2a Phase 2a interim data due 2Q 2019.
$517.4 million

AXSM – Axsome Therapeutics Inc.
AXS-05 ASCEND
Major Depressive Disorder

Phase 2 Phase 2 data met primary endpoint - January 7, 2019.
$272 million

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111)
Waldenström’s macroglobulinemia

Phase 1/2 Phase 1 updated data due 1H 2019.
$7.9 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111)
Relapsed or refractory mantle cell lymphoma (MCL)

Phase 2 Phase 2 updated data due 1H 2019.
$7.9 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111) vs Ibrutinib
Refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)

Phase 3 Phase 3 data due 2H 2019.
$7.9 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111) with Bendamustine Plus Rituximab
First-line chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)

Phase 3 Phase 3 data from 17p del cohort due 2H 2019.
$7.9 billion

BGNE – BeiGene Ltd.
Tislelizumab
Hepatocellular carcinoma (HCC) - second-line

Phase 2 Phase 2 data due 2019.
$7.9 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111)
Waldenström’s macroglobulinemia

Phase 3 Phase 3 data to be presented 1H 2019.
$7.9 billion

BLPH – Bellerophon Therapeutics Inc.
INOpulse delivery device
Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)

Phase 2b Phase 2b top-line data released January 7, 2019 with Cohort 2 and 3 data due later in 2019.
$39.3 million

BLUE – bluebird bio Inc.
bb2121 - KarMMa
Relapsed/refractory multiple myeloma

Phase 2 Phase 2 data due 2H 2019.
$6.5 billion

BMRN – BioMarin Pharmaceutical Inc.
Vosoritide
Achondroplasia

Phase 3 Phase 3 top-line data due end of 2019.
$16.7 billion

BOLD – Audentes Therapeutics Inc.
AT342 - VALENS
Crigler-Najjar

Phase 1/2 Phase 1/2 updated data due 1Q 2019.
$990 million

BOLD – Audentes Therapeutics Inc.
AT132 - ASPIRO
X-Linked Myotubular Myopathy

Phase 1/2 Next update at American Society of Gene and Cell Therapy (ASGCT) - April 29-May 2, 2019.
$990 million

BSTC – BioSpecifics Technologies Corp
XIAFLEX (collagenase clostridium histolyticum) - RELEASE
Edematous fibrosclerotic panniculopathy (“cellulite”)

BLA Filing BLA filing 2H 2019.
$452.6 million

CALA – Calithera Biosciences Inc.
CB-839 + AFINITOR (everolimus)‎ - ENTRATA
Clear cell renal cell carcinoma

Phase 2 Phase 2 top-line data due 2H 2019.
$156.3 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) injection - KALM-1
Chronic kidney disease (CKD) on hemodialysis (HD) with Moderate-to Severe Pruritus

Phase 3 Phase 3 data due 2Q 2019.
$592.5 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) injection - KALM-2 global
Chronic kidney disease (CKD) on hemodialysis (HD) with Moderate-to Severe Pruritus

Phase 3 Phase 3 data due 2H 2019.
$592.5 million

CELG – Celgene Corporation
OTEZLA
Scalp Psoriasis

sNDA Filing sNDA filing due 2Q 2019.
$60.6 billion

CELG – Celgene Corporation
ABRAXANE - PANC-003 apact
Adjuvant Therapy in Surgically Resected Pancreatic Cancer

Phase 3 Phase 3 data due 2019.
$60.6 billion

CELG – Celgene Corporation
REVLIMID - AUGMENT
Relapsed or Refractory Follicular Lymphoma

sNDA Filing sNDA filed.
$60.6 billion

CELG – Celgene Corporation
REVLIMID - ROBUST
First-line ABC Diffuse large B-cell lymphoma (DLBCL)

Phase 3 Phase 3 data due 2019.
$60.6 billion

CELG – Celgene Corporation
OTEZLA
Behçet’s Disease

PDUFA PDUFA date for sNDA July 21, 2019.
$60.6 billion

CELG – Celgene Corporation
TECENTRIQ (atezolizumab) plus Abraxane
Triple-negative breast cancer

PDUFA PDUFA date March 12, 2019.
$60.6 billion

CELG – Celgene Corporation
Ozanimod - TRUE NORTH
Ulcerative Colitis

Phase 3 Phase 3 enrolling. Enrolment to be completed 1H 2019.
$60.6 billion

CELG – Celgene Corporation
Ozanimod - RADIANCE
Relapsing Multiple Sclerosis

NDA Filing Refusal-to-file letter issued February 27, 2018. NDA to be refiled 1Q 2019.
$60.6 billion

CELG – Celgene Corporation
Lico-cel (CD-19 JCAR017) - TRANSCEND
r/r B cell non-Hodgkin lymphoma - cancer

Phase 1/2 Pivotal data due 2019.
$60.6 billion

CFRX – ContraFect Corporation
CF-301
Serious infections caused byStaph aureus including MRSA

Phase 2 Phase 2 trial noted clinically meaningful data but not statistically significant.
$41.3 million

CKPT – Checkpoint Therapeutics Inc.
CK-301
Lung and other solid tumors

Phase 1 Phase 1 initiation announced January 8, 2019 with initial data likely 1H 2019.
$110 million

CNST – Constellation Pharmaceuticals Inc.
CPI-1205 - ProSTAR
Castration-resistant prostate cancer

Phase 1/2 Phase 1b data 2Q 2019. Phase 2 interim data due 2H 2019.
$160.1 million

CYAD – Celyad SA
CYAD-01 and FOLFOX - SHRINK
Colorectal cancer

Phase 1 Phase 1 data due 2019.
$271.7 million

CYAD – Celyad SA
CYAD-01 - THINK
Various cancers - solid and hematologic cancers

Phase 1/2 Phase 1 additional data due 1H 2019.
$271.7 million

CYCC – Cyclacel Pharmaceuticals Inc.
Sapacitabine in combination with olaparib
BRCA mutant breast cancer

Phase 1/2 Phase 1b/2 initial data due 2019.
$9.1 million

CYCC – Cyclacel Pharmaceuticals Inc.
CYC140
Leukemias

Phase 1 Phase 1 initial data due 2019.
$9.1 million

CYCC – Cyclacel Pharmaceuticals Inc.
CYC065 + venetoclax
Chronic lymphocytic leukemia (CLL)

Phase 1 Phase 1 initial data due 2019.
$9.1 million

DARE – Dare Bioscience Inc.
DARE-HRT1
Hormone replacement therapy

Phase 1 Phase 1 top-line data due 2H 2019.
$9.5 million

DARE – Dare Bioscience Inc.
Sildenafil Cream, 3.6%
Female Sexual Arousal Disorder (FSAD)

Phase 2 Phase 2b trial to be initiated in 2019 with data due 4Q 2020.
$9.5 million

DARE – Dare Bioscience Inc.
DARE-BV1
Bacterial Vaginosis

Phase 3 Phase 3 trial to be initiated 2H 2019.
$9.5 million

DOVA – Dova Pharmaceuticals Inc.
Avatrombopag
Chemotherapy-induced thrombocytopenia (CIT)

Phase 3 Phase 3 data due 1H 2020.
$212.1 million

DRRX – DURECT Corporation
DUR-928
Nonalcoholic steatohepatitis (NASH)

Phase 1b Phase 2 trial to be initiated 1Q 2019 with data due 2H 2019.
$93.5 million

EDIT – Editas Medicine Inc.
EDIT-101
Leber Congenital Amaurosis type 10 (LCA10).

Phase 1/2 Phase 1/2 trial to be initiated 2H 2019.
$961 million

ENTA – Enanta Pharmaceuticals Inc.
EDP-305 ARGON-1
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 initial data due 3Q 2019.
$1.5 billion

ENTA – Enanta Pharmaceuticals Inc.
EDP-938
Respiratory Syncytial Virus

Phase 2 Phase 2 preliminary data due mid-2019.
$1.5 billion

EVFM – Evofem Biosciences Inc.
Amphora - AMPREVENCE
Prevention of urogenital Chlamydia trachomatis infection in women

Phase 2/3 Phase 2b data due 4Q 2019.
$99.6 million

EVFM – Evofem Biosciences Inc.
Amphora - AMPOWER
contraceptive vaginal gel

NDA Filing Phase 3 data released December 17, 2018. Primary endpoint met. NDA to be refiled 2Q 2019.
$99.6 million

FGEN – FibroGen Inc
FG-3019
Duchenne muscular dystrophy

Phase 2 Phase 2 initial data due late 1Q 2019.
$4.4 billion

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

Phase 3 Pooled MACE safety data 1H 2019.
$4.4 billion

FGEN – FibroGen Inc
Pamrevlumab (FG-3019)
Idiopathic pulmonary fibrosis

Phase 2 Phase 3 trial to commence 2019.
$4.4 billion

FOLD – Amicus Therapeutics Inc.
CLN6 Gene Therapy
Batten disease

Phase 1/2 Phase 1/2 preliminary data due mid-2019.
$2.2 billion

GEMP – Gemphire Therapeutics Inc.
Gemcabene
Familial Partial Lipodystrophy (FPL)

Phase 2a Phase 2 top-line data due 2Q 2019.
$15.7 million

GERN – Geron Corporation
Imetelstat - IMerge Study
Myelodysplastic syndromes

Phase 2/3 Phase 3 trial to commence mid-2019.
$188.2 million

GLPG – Galapagos NV
GLPG1690 - NOVESA
Systemic sclerosis

Phase 2 Phase 2 initiation announced January 7, 2019.
$5.6 billion

GMDA – Gamida Cell Ltd.
NiCord
Acute myeloid leukemia

Phase 3 Phase 3 top-line data due 1H 2020.
$278.3 million

GMDA – Gamida Cell Ltd.
NiCord
Severe aplastic anemia

Phase 1/2 Phase 1/2 preliminary data due 1H 2019.
$278.3 million

GMDA – Gamida Cell Ltd.
NAM-NK
Non-Hodgkin lymphoma; Multiple myeloma

Phase 1 Phase 1 data due 1H 2019.
$278.3 million

GNCA – Genocea Biosciences Inc.
GEN-009 vaccine
Various cancers

Phase 1/2 Phase 1/2 initial immunogenicity data due mid-2019.
$50.2 million

GWPH – GW Pharmaceuticals Plc
Epidiolex
Tuberous Sclerosis Complex

Phase 3 Phase 3 data due 2Q 2019.
$4.1 billion

GWPH – GW Pharmaceuticals Plc
Sativex
Spasticity due multiple sclerosis

Phase 2 Phase 3 trial planned for 2H 2019.
$4.1 billion

HZNP – Horizon Pharma plc
Teprotumumab
Thyroid Eye Disease (TED)

Phase 3 Phase 3 data due 1Q 2019.
$3.7 billion

ICPT – Intercept Pharmaceuticals Inc.
Ocaliva (Obeticholic acid (OCA)) - REGENERATE
Adult nonalcoholic steatohepatitis (NASH) patients.

Phase 3 Phase 3 data due 1Q 2019.
$3.3 billion

ICPT – Intercept Pharmaceuticals Inc.
Ocaliva - Obeticholic acid (OCA) - REVERSE
NASH patients with compensated cirrhosis

Phase 3 Phase 3 enrolment to be completed in 2019.
$3.3 billion

IDRA – Idera Pharmaceuticals Inc.
Tilsotolimod + ipilimumab - ILLUMINATE 204
Cancer - melanoma

Phase 2 Phase 2 final data due 2H 2019.
$63.3 million

IDRA – Idera Pharmaceuticals Inc.
Tilsotolimod (IMO-2125) + ipilimumab - ILLUMINATE 301
Melanoma

Phase 3 Phase 3 completion of enrolment due YE 2019.
$63.3 million

IRWD – Ironwood Pharmaceuticals Inc.
IW-1973
Diabetic nephropathy

Phase 2 Phase 2 data due 2H 2019.
$1.9 billion

IRWD – Ironwood Pharmaceuticals Inc.
MD-7246
Irritable Bowel Syndrome

Phase 2 Phase 2 trial to be initiated 1Q 2019.
$1.9 billion

IRWD – Ironwood Pharmaceuticals Inc.
IW-1973
Heart failure HFpEF

Phase 2 Phase 2 data due 2H 2019.
$1.9 billion

IRWD – Ironwood Pharmaceuticals Inc.
Linaclotide
Irritable Bowel Syndrome with Constipation (IBS-C)

Phase 3 Phase 3b data due mid-2019.
$1.9 billion

IRWD – Ironwood Pharmaceuticals Inc.
Olinciguat (IW-1701) - STRONG-SCD
Sickle Cell disease

Phase 2 Phase 2 data due 2H 2019.
$1.9 billion

KPTI – Karyopharm Therapeutics Inc.
Selinexor - SEAL
Dedifferentiated liposarcoma

Phase 2/3 Phase 3 top-line data due end of 2019.
$542.7 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor - BOSTON
Multiple myeloma

Phase 3 Phase 3 data due end of 2019.
$542.7 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor - SADAL
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)

NDA Filing NDA filing due 2019.
$542.7 million

KURA – Kura Oncology Inc.
KO-947
Solid tumors

Phase 1 Phase 1 data due 2019.
$582.7 million

LJPC – La Jolla Pharmaceutical Company
LJPC-401
Beta thalassemia patients suffering from iron overload

Phase 3 Pivotal data due mid-2020.
$157.1 million

LJPC – La Jolla Pharmaceutical Company
LJPC‑401
Hereditary Hemochromatosis

Phase 2 Phase 2 data due 4Q 2019.
$157.1 million

LQDA – Liquidia Technologies Inc.
LIQ861 - INSPIRE
Pulmonary arterial hypertension

Phase 3 Phase 3 trial is enrolling. PK data due 2Q.
$228 million

MGTA – Magenta Therapeutics Inc.
MGTA-145
Healthy volunteers

Phase 1 Phase 1 initial data due at ASH 2019.
$231.7 million

MGTA – Magenta Therapeutics Inc.
MGTA-456
Acute Lymphoblastic Leukemia

Phase 2 Phase 2 data due at ASH 2019.
$231.7 million

NBIX – Neurocrine Biosciences Inc.
Elagolix
Uterine Fibroids

NDA Filing NDA filing due 2019.
$8.1 billion

NBIX – Neurocrine Biosciences Inc.
Opicapone
Parkinson's disease

NDA Filing NDA filing planned for 2Q 2019.
$8.1 billion

NBIX – Neurocrine Biosciences Inc.
NBI-74788
Congenital Adrenal Hyperplasia (CAH) - adults

Phase 2a Phase 2a data due 1Q 2019.
$8.1 billion

NBIX – Neurocrine Biosciences Inc.
NBI-74788
Congenital Adrenal Hyperplasia (CAH) - children

Phase 2 Phase 2 initiation 2Q/3Q 2019.
$8.1 billion

NOVN – Novan Inc.
SB206
Molluscum contagiosum skin infection

Phase 3 Phase 3 trial to be initiated 1H 2019 with data due late 2019 or early 2020.
$38.8 million

NTEC – Intec Pharma Ltd.
AP-CDLD
Advanced Parkinson's Disease

Phase 3 Phase 3 data due mid-2019.
$243.9 million

NVCR – NovoCure Limited
Tumor Treating Fields (TTFields) LUNAR
Non-small cell lung cancer (NSCLC)

Phase 3 Phase 3 interim data 2020. Final data 2021.
$4.4 billion

NVCR – NovoCure Limited
Tumor Treating Fields (TTFields) - INNOVATE
Ovarian cancer

Phase 3 Phase 3 interim analysis 2022.
$4.4 billion

NVCR – NovoCure Limited
Tumor Treating Fields (TTFields) - HEPANOVA
Liver cancer

Phase 2 Phase 2 data due 2020.
$4.4 billion

NVCR – NovoCure Limited
Tumor Treating Fields (TTFields) - PANOVA 3
Pancreatic cancer

Phase 3 Phase 3 interim analysis 2021.
$4.4 billion

NVCR – NovoCure Limited
Tumor Treating Fields (TTFields) METIS
Brain Metastases Originating from Non-Small Cell Lung Cancer

Phase 3 Phase 3 data due 2021.
$4.4 billion

RCUS – Arcus Biosciences Inc.
AB928 + AB122
Solid tumors

Phase 1 Phase 1 data due June 2019.
$419.2 million

RCUS – Arcus Biosciences Inc.
AB928 + anti-PD-1 therapy + chemotherapy
Non-small cell lung cancer

Phase 1 Phase 1 data due June 2019.
$419.2 million

RCUS – Arcus Biosciences Inc.
AB928
Solid tumors

Phase 1 Phase 1/2 data from the dose-escalation portion due June 2018.
$419.2 million

REGN – Regeneron Pharmaceuticals Inc.
REGN1979
Follicular Lymphoma

Phase 2 Phase 2 trial to commence 1H 2019.
$43.4 billion

RIGL – Rigel Pharmaceuticals Inc.
Fostamatinib
Autoimmune hemolytic anemia (AIHA)

Phase 2 Phase 3 trial to commence 1H 2019.
$333.3 million

RTRX – Retrophin Inc.
RE-024 fosmetpantotenate
Pantothenate kinase-associated neurodegeneration (PKAN)

Phase 3 Phase 3 top-line data due 3Q 2019.
$865 million

RTRX – Retrophin Inc.
CNSA-001
Phenylketonuria (PKU)

Phase 2 Phase 2 data due 1H 2019.
$865 million

RTRX – Retrophin Inc.
Sparsentan
IgA nephropathy

Phase 3 Phase 3 data due 1H 2022.
$865 million

RTRX – Retrophin Inc.
Sparsentan - DUPLEX
Focal segmental glomerulosclerosis (FSGS)

Phase 3 Phase 3 top-line data due 2H 2020.
$865 million

RUBY – Rubius Therapeutics Inc.
RTX-134
Phenylketonuria (PKU)

Phase 1/2 Phase 1b/2 initial data due 2H 2019.
$1.1 billion

RVNC – Revance Therapeutics Inc.
RT002
Moderate to severe glabellar (frown) lines

BLA Filing BLA filing due 1H 2019.
$635.4 million

RVNC – Revance Therapeutics Inc.
RT002
Plantar fasciitis

Phase 2a Phase 2 completion of enrolment due 2H 2019.
$635.4 million

RVNC – Revance Therapeutics Inc.
RT002
Upper limb spasticity

Phase 2 Phase 2 completion of enrolment due 2H 2019.
$635.4 million

RVNC – Revance Therapeutics Inc.
RT002 - ASPEN
Cervical dystonia

Phase 3 Phase 3 completion of enrolment due early 2020.
$635.4 million

SAGE – Sage Therapeutics Inc.
SAGE-217
Major Depressive Disorder (MDD)

Phase 3 Phase 3 data due 2020.
$6.1 billion

SAGE – Sage Therapeutics Inc.
SAGE-217
Bipolar depression

Phase 2 Phase 2 Part A data due 1H 2019.
$6.1 billion

SAGE – Sage Therapeutics Inc.
SAGE-324
Essential tremor

Phase 1 Phase 1 data due 1H 2019.
$6.1 billion

SAGE – Sage Therapeutics Inc.
SAGE-217
Postpartum depression (PPD)

Phase 3 Phase 3 data met primary endpoint - January 7, 2019.
$6.1 billion

SAGE – Sage Therapeutics Inc.
SAGE-718
Huntington's disease

Phase 1 Phase 1 data due 2H 2019.
$6.1 billion

SAGE – Sage Therapeutics Inc.
SAGE-217
Insomnia

Phase 3 Phase 3 data due 2020.
$6.1 billion

SLGL – Sol-Gel Technologies Ltd.
TWIN
Acne

Phase 3 Phase 3 data due 4Q 2019.
$110.1 million

SLS – SELLAS Life Sciences Group Inc.
Galinpepimut-S and Keytruda (pembrolizumab)
Solid tumors

Phase 1/2 Phase 1/2 interim analysis due 4Q 2019.
$32.6 million

SLS – SELLAS Life Sciences Group Inc.
Galinpepimut-S
Acute Myeloid Leukemia (AML)

Phase 3 Phase 3 trial planned for 2Q 2019 with interim analysis 3Q 2020.
$32.6 million

SNDX – Syndax Pharmaceuticals Inc.
SNDX-6352
Chronic graft versus host disease

Phase 1 Phase 1 initial data due 2H 2019.
$128.7 million

SNDX – Syndax Pharmaceuticals Inc.
E2112
HR+, HER2- breast cancer

Phase 3 Phase 3 PFS date did not meet primary endpoint - October 25, 2018. OS data due 2Q 2019.
$128.7 million

SNDX – Syndax Pharmaceuticals Inc.
Entinostat plus Tecentriq - ENCORE 603
Ovarian cancer

Phase 1/2 Phase 2 top-line data due 1Q 2019.
$128.7 million

SNDX – Syndax Pharmaceuticals Inc.
Entinostat plus Tecentriq - ENCORE 602
Triple negative breast cancer (TNBC)

Phase 2 Phase 2 top-line data due 2Q 2019.
$128.7 million

SPPI – Spectrum Pharmaceuticals Inc.
Poziotinib
Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2 Phase 2 top-line data due 2H 2019.
$1.1 billion

SURF – Surface Oncology Inc.
NZV930
Solid tumors

Phase 1 Phase 1 data likely 2019.
$134 million

TBPH – Theravance Biopharma Inc.
TD-9855
nOH

Phase 3 Phase 3 dosing to commence early 2019.
$1.5 billion

TORC – resTORbio Inc.
RTB101
Parkinson's disease

Phase 2 Phase 2 trial to be initiated 1Q 2019.
$231.5 million

TORC – resTORbio Inc.
RTB101 and Everolimus
Respiratory Tract Infections

Phase 2b Phase 3 trial to be initiated 1H 2019.
$231.5 million

XENE – Xenon Pharmaceuticals Inc.
XEN1101
Adult focal seizures

Phase 2 Phase 2 data due 2H 2020.
$194.9 million

XENE – Xenon Pharmaceuticals Inc.
XEN496
Epilepsy

Phase 3 Phase 3 trial to be initiated following IND filing 3Q 2019.
$194.9 million

XLRN – Acceleron Pharma Inc.
Luspatercept
Myelofibrosis

Phase 2 Phase 2 data 2H 2019.
$1.9 billion

XLRN – Acceleron Pharma Inc.
ACE-083
Charcot-Marie-Tooth disease (CMT)

Phase 2 Phase 2 Part 2 preliminary data due by the end of 2019.
$1.9 billion

XLRN – Acceleron Pharma Inc.
Sotatercept
Pulmonary arterial hypertension (PAH)

Phase 2 Phase 2 data due 1H 2020.
$1.9 billion

XLRN – Acceleron Pharma Inc.
ACE-083
Facioscapulohumeral muscular dystrophy

Phase 2 Phase 2 Part 2 data due 2H 2019.
$1.9 billion

XLRN – Acceleron Pharma Inc.
ACE-2494
Healthy volunteers

Phase 1 Preliminary results due 1H 2019.
$1.9 billion

XLRN – Acceleron Pharma Inc.
Luspatercept - BEYOND
Non-transfusion-dependent beta-thalassemia

Phase 2 Phase 2 data due 2019.
$1.9 billion

ZYME – Zymeworks Inc.
ZW25
HER2-expressing Cancers - ovarian, breast, gastric

Phase 1 Phase 1 updated data due 2019. Phase 2 trial to be initiated 2019.
$504.9 million